Lumacaftor alone and combined with ivacaftor: preclinical and clinical trial experience of F508del CFTR correction

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

(2015) Claire E. Wainwright et al.   NEW ENGLAND JOURNAL OF MEDICINE

Ivacaftor for the treatment of patients with cystic fibrosis and the G551D mutation: a systematic review and cost-effectiveness analysis

(2014) Penny Whiting et al.   HEALTH TECHNOLOGY ASSESSMENT

Cystic fibrosis: An inherited disease affecting mucin-producing organs

(2014) Camille Ehre et al.   INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY

Impaired mucus detachment disrupts mucociliary transport in a piglet model of cystic fibrosis

(2014) M. J. Hoegger et al.   SCIENCE

CFTR Potentiators: Not an Open and Shut Case

(2014) J. P. Clancy   Science Translational Medicine

Some gating potentiators, including VX-770, diminish  F508-CFTR functional expression

(2014) G. Veit et al.   Science Translational Medicine

Potentiator ivacaftor abrogates pharmacological correction of  F508 CFTR in cystic fibrosis

(2014) D. M. Cholon et al.   Science Translational Medicine

Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial

(2014) Eitan Kerem et al.   Lancet Respiratory Medicine

A CFTR corrector (lumacaftor) and a CFTR potentiator (ivacaftor) for treatment of patients with cystic fibrosis who have a phe508del CFTR mutation: a phase 2 randomised controlled trial

(2014) Michael P Boyle et al.   Lancet Respiratory Medicine

Lung Clearance Index as an Outcome Measure for Clinical Trials in Young Children with Cystic Fibrosis. A Pilot Study Using Inhaled Hypertonic Saline

(2013) Padmaja Subbarao et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Efficacy and Safety of Ivacaftor in Patients Aged 6 to 11 Years with Cystic Fibrosis with aG551DMutation

(2013) Jane C. Davies et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Editorial (Hot Topic:Cystic Fibrosis: CFTR Modulators and their Mechanism of Action)

(2013) Erika Nieddu   CURRENT PHARMACEUTICAL DESIGN

Intestinal CFTR expression alleviates meconium ileus in cystic fibrosis pigs

(2013) David A. Stoltz et al.   JOURNAL OF CLINICAL INVESTIGATION

Normal mucus formation requires cAMP-dependent HCO3−secretion and Ca2+-mediated mucin exocytosis

(2013) Ning Yang et al.   JOURNAL OF PHYSIOLOGY-LONDON

A functional CFTR assay using primary cystic fibrosis intestinal organoids

(2013) Johanna F Dekkers et al.   NATURE MEDICINE

Airway inflammation in cystic fibrosis: molecular mechanisms and clinical implications

(2013) Malena Cohen-Cymberknoh et al.   THORAX

Ivacaftor in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

(2012) Patrick A. Flume et al.   CHEST

Molecular organization of the mucins and glycocalyx underlying mucus transport over mucosal surfaces of the airways

(2012) M Kesimer et al.   Mucosal Immunology

A CFTR Potentiator in Patients with Cystic Fibrosis and theG551DMutation

(2011) Bonnie W. Ramsey et al.   NEW ENGLAND JOURNAL OF MEDICINE

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

(2011) F. Van Goor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation

(2011) J P Clancy et al.   THORAX

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

(2011) Gergely L. Lukacs et al.   TRENDS IN MOLECULAR MEDICINE

Role of epithelial HCO3− transport in mucin secretion: lessons from cystic fibrosis

(2010) Paul M. Quinton   AMERICAN JOURNAL OF PHYSIOLOGY-CELL PHYSIOLOGY

Loss of Cystic Fibrosis Transmembrane Conductance Regulator Function Produces Abnormalities in Tracheal Development in Neonatal Pigs and Young Children

(2010) David K. Meyerholz et al.   AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE

Loss of Anion Transport without Increased Sodium Absorption Characterizes Newborn Porcine Cystic Fibrosis Airway Epithelia

(2010) Jeng-Haur Chen et al.   CELL

Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation

(2010) Frank J. Accurso et al.   NEW ENGLAND JOURNAL OF MEDICINE

Functional Stability of Rescued ΔF508 Cystic Fibrosis Transmembrane Conductance Regulator in Airway Epithelial Cells

(2009) Asta Jurkuvenaite et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Pharmaceuticals Targeting Nonsense Mutations in Genetic Diseases

(2009) Steven M. Rowe et al.   BIODRUGS

BAP31 Interacts with Sec61 Translocons and Promotes Retrotranslocation of CFTRΔF508 via the Derlin-1 Complex

(2008) Bing Wang et al.   CELL

Cystic fibrosis: impaired bicarbonate secretion and mucoviscidosis

(2008) Paul M Quinton   LANCET